Protein Aggregation Inhibitor Development

Protein aggregation inhibitors work on the mechanisms which are responsible for protein aggregation, with the objective of maintaining normal structural and functional properties of the proteins. Protheragen works to develop effective solutions for the inhibition of protein aggregation in rare neurological diseases. In collaboration with other researchers in the area, Protheragen makes available the services and expertise necessary to conduct high-level scientific research.

Overview of Protein Aggregation Inhibitors for Rare Neurological Diseases

The aggregation of protein is a defining feature of numerous neurodegenerative and movement disorders, including Alzheimer's disease (AD), Parkinson's disease (PD), amyotrophic lateral sclerosis (ALS), and Huntington's disease (HD). These disorders are marked by the progression of misfolded proteins into cytotoxic oligomers, fibrils, and insoluble plaques that lead to neuronal cell dysfunction and death. Inhibitors of protein aggregation offer potential therapeutics aimed at disrupting these pathological processes.

Fig.1 Six main ways of protein aggregates damage the metabolism of cells. (Wen J H, et al., 2023)

Mechanisms of Action of Protein Aggregation Inhibitors

Protein aggregation inhibitors prevent misfolded proteins from accumulating in a pathological manner by one of three possible ways: stabilizing the native protein structure, eradicating toxic oligomers or fibrils, and augmenting cellular clearance mechanisms. These inhibitors manifest in various forms, including but not limited to, monoclonal antibodies, small molecules, peptides, and new therapies such as PROTACs. Through targeted intervention at various different stages of the aggregation cascade, these inhibitors offer a tailored approach towards the treatment of neurodegenerative disorders.

Fig.2 Types and mechanisms of action of conformational inhibitors of protein aggregation. (Ramírez M C F, et al., 2023)

Development of Protein Aggregation Inhibitors for Rare Neurological Diseases

Disclaimer: Protheragen focuses on providing preclinical research services. This table is for information exchange purposes only. This table is not a treatment plan recommendation. For guidance on treatment options, please visit a regular hospital.

Our Services

At Protheragen, we specialize in developing end-to-end protein aggregation inhibitors for rare neurological disorders. We employ innovative disease models alongside preclinical studies to expedite therapeutic breakthroughs. Our work spans from target validation to the high-throughput screening of lead compounds. Incorporating CNS-centric biomarker translation into drug design, we offer pertinent drug candidate development for Aβ, tau, SOD1, and FUS aggregates, addressing the significant unmet needs in neurodegenerative disorders.

Diverse Development Platforms for Protein Aggregation Inhibitors

• Small Molecule Drug Development
• Cell Therapy Development
• Gene Therapy Development

• Therapeutic Antibody Development
• Therapeutic Peptide Development
• Therapeutic Protein Development

Workflow of Protein Aggregation Inhibitor Development

Protheragen develops custom solutions, leveraging deep industry expertise and the latest technologies, to help expedite research processes while assisting in the advancement of protein aggregation inhibitors. If you are interested in our services, please feel free to contact us for more details and quotation information of related services.

References

• Wen J H, He X H, Feng Z S, et al. Cellular protein aggregates: formation, biological effects, and ways of elimination[J]. International journal of molecular sciences, 2023, 24(10): 8593.
• Ramírez M C F, Afrin S, Saelices L. Conformational inhibitors of protein aggregation[J]. Current Opinion in Structural Biology, 2023, 83: 102700.

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